New Breakthroughs Offer New Hope

In November 2014, scientists at Thomas Jefferson University announced a new breakthrough in ALS treatments -- blocking certain types of proteins in the brain may help drugs that have so far failed to treat ALS become effective. In our brains, there are proteins that remove toxins and pump them out through the lymph system. In ALS patients, however, these protein pumps work in overdrive, pumping the ALS drugs out of the brain as well, which may be why a majority of ALS drugs fail to be effective, despite promising early-stage research.

There is currently only one FDA-approved medication for ALS: riluzole, which may be able to extend the life of an ALS patient by a few months because it delays the period before a patient would need to be put on a ventilator. This drug has only been found to delay the progression of ALS and does not appear to repair the damage that has already been done to the motor neurons.

In a trial conducted on mice infected with ALS, the Thomas Jefferson University researchers were able to identify two specific “pumps” in the brain that interact with riluzole and limit its effectiveness: P-glycoprotein (P-gp) and breast-cancer resistant protein. When treated with an experimental compound called Elacridar to block these proteins, researchers found that the mice had an extended lifespan, and some of the symptoms were alleviated, including improving and preserving muscle strength.

One of the findings, published in the Annals of Clinical and Translational Neurology, indicated that previously discarded ALS drugs may be worth revisiting in combination with Elacridar. They concluded that the reason many ALS drugs seem to fail could be a result of the brain’s own defense mechanisms. As the disease increasingly attacks and damages motor neurons, the brain creates more pumps to try to fight back and remove the damaged neurons while also removing the ALS drugs introduced to fight the disease.

While drug treatments may be re-evaluated in combination with selective pump inhibitors, research abroad has led to a different approach in battling ALS that is also showing promise: stem cell transplants.